Methods and compositions for cns delivery of iduronate-2-sulfatase
AssigneeTakeda Pharmaceutical Company Limited
InventorsGaozhong Zhu, Kris Lowe, Zahra Shahrokh, James Christian, Richard Fahrner, Jing Pan, Teresa Leah Wright, Pericles Calias
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Abstract
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.
Claims31
A stable formulation for direct administration to the cerebrospinal fluid (CSF) comprising a recombinant iduronate-2-sulfatase (I2S) protein at a concentration of at least 10 mg/mL, a salt, a polysorbate surfactant, and no greater than 5 mM phosphate.
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The method of 41, wherein the direct administration of the formulation to the CSF results in lysosomal localization in brain target tissues, spinal cord neurons and/or peripheral target tissues.
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Dependencies are inferred from the claim text.